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Register of orphan medicinal products


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Adeno-associated viral vector serotype 9 containing the human HGSNAT gene

EU orphan designation number: EU/3/15/1491   
Active ingredient: Adeno-associated viral vector serotype 9 containing the human HGSNAT gene
Indication: Treatment of mucopolysaccharidosis IIIC (Sanfilippo C syndrome)
Sponsor: Cochamo Systems Ltd
104 Manchester Road, Manchester, M27 5FQ, United Kingdom

   Public summary of scientific opinion    

European Commission proceduresGoto top of the page

Close date procedure Procedure type EMEA number Decision summary publ decision docs annex
27/05/2015 Orphan designation EMA/OD/322/14 (2015)3571 of 21/05/2015